CUDC-907 for Pediatric Cancer

Not currently recruiting at 3 trial locations

Overseen By David S Shulman, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Dana-Farber Cancer Institute

Must not be taking: Corticosteroids, NSAIDs, Anticoagulants, Heparins

Disqualifiers: Pregnancy, Breastfeeding, Diabetes, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 2 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, CUDC-907, to determine its effectiveness in treating hard-to-treat pediatric cancers such as solid tumors, lymphomas, and brain tumors. The aim is to assess whether this drug can combat these cancers when other treatments have failed. Children with relapsed or resistant forms of these cancers, who have not responded to other treatments, may be suitable candidates for this trial. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop all current medications, but you cannot take certain drugs like systemic corticosteroids (unless for specific conditions), non-steroidal anti-inflammatory drugs, oral anticoagulants, and therapeutic heparins. It's best to discuss your current medications with the study team to see if they are allowed.

Is there any evidence suggesting that CUDC-907 is likely to be safe for humans?

Research has shown that CUDC-907 has been tested for safety in adults with blood cancers and was generally well-tolerated. The treatment was administered for five consecutive days, followed by a two-day break, which proved effective. Another study confirmed the safety of CUDC-907, allowing further research. While these results are promising, it is important to note that this trial is still in the early stages of testing for children's cancer. Researchers are still assessing its safety for children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CUDC-907 for pediatric cancer because it targets cancer cells differently than existing treatments. Most treatments for pediatric cancer, like chemotherapy, attack rapidly dividing cells, which can affect both cancerous and healthy cells. CUDC-907 combines an HDAC inhibitor and a PI3K inhibitor, specifically targeting cancer cell growth and survival pathways while potentially sparing healthy cells. This dual action may offer a more precise attack on tumor cells, reducing side effects and improving outcomes for young patients. Additionally, its oral administration provides a more convenient and less invasive option compared to many traditional therapies.

What evidence suggests that CUDC-907 might be an effective treatment for pediatric cancer?

Research has shown that CUDC-907, the investigational treatment in this trial, targets two key proteins in cancer cells, HDAC and PI3K, which aid cancer growth. Studies have found that CUDC-907 can slow the growth of various cancers, including brain cancer (glioblastoma), prostate cancer, and leukemia. Early research, conducted before human testing, suggested that blocking these proteins can reduce tumor growth in different cancers. CUDC-907's dual action on both proteins has shown promising results, offering hope for treating difficult childhood cancers. ^{1 2 3 6 7}

Who Is on the Research Team?

David S Shulman, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

This trial is for children and young adults aged 1 to 21 with relapsed or refractory solid tumors, CNS tumors, or lymphoma. They must have a performance status of at least 50%, measurable disease, normal organ function, be able to swallow medication without chewing, and not be pregnant or breastfeeding. Participants need stable neurological conditions and can't be on certain medications like corticosteroids (unless for specific diseases), NSAIDs, anticoagulants, or have uncontrolled illnesses.

Inclusion Criteria

Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy

Patients must have normal organ function as defined

Patients must be able to swallow either intact capsules or mini-tabs without chewing

Exclusion Criteria

Patients must not be receiving non-steroidal anti-inflammatory drugs, oral anticoagulants, and therapeutic heparins

Pregnant participants will not be entered on this study

Breastfeeding mothers are not eligible

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive CUDC-907 orally once daily for 5 consecutive days per week followed by two days without dosing. Dose escalation follows a standard 3+3 design.

Up to 2 years

Weekly visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CUDC-907

Trial Overview The study is testing CUDC-907's effectiveness in treating pediatric patients who haven't responded well to standard treatments for various types of cancer including neuroblastoma and brain tumors. It aims to find out if this new drug can help where other treatments haven't worked.

How Is the Trial Designed?

Group I: CUDC-907 Experimental Treatment 1 Intervention

* CUDC-907 orally administered * CUDC-907 once daily for 5 consecutive days per week followed by two days without dosing * Dose level assigned at registration * Pre-dose pharmacokinetic blood sample will be collected * Dose escalation will follow a standard 3+3 design

Who Is Running the Clinical Trial?

Dana-Farber Cancer Institute

Lead Sponsor

Trials

1,128

Recruited

382,000+

Curis, Inc.

Industry Sponsor

Trials

17

Recruited

1,100+

Published Research Related to This Trial

Out of 236 Individual Case Safety Reports (ICSRs) analyzed, only 18 (7.6%) were classified as off-label cases, indicating a low incidence of off-label use of antineoplastic drugs in pediatric patients, primarily related to therapeutic indications.

Among the off-label cases, 29 adverse drug reactions (ADRs) were reported, with common reactions including diarrhea and neutropenia, suggesting that while off-label use is limited, it can still lead to significant safety concerns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System. Mascolo, A., Scavone, C., Bertini, M., et al.[2022]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8870466/

Dual Targeting of PI3K and HDAC by CUDC-907 Inhibits ... CUDC-907 has been reported to inhibit pancreatic adenocarcinoma [35], glioblastoma [36], prostate cancer [21], and acute myeloid leukemia [20] ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02909777?term=AREA%5BSponsorSearch%5D(COVERAGE%5BFullMatch%5DEXPANSION%5BNone%5D(%22Curis,%20Inc.%22))&rank=4

Trial of CUDC-907 in Children and Young Adults With ... CUDC-907 is an oral drug that blocks certain proteins in tumor cells. These proteins may be important in the growth of some cancers.

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2017.35.15_suppl.TPS10576

Phase 1 multicenter trial of CUDC-907 in children and ... Preclinical data demonstrate that inhibition of these enzymes decreases tumor growth across a range of histologies. Data from preclinical and ...

4. nature.com nature.com/articles/s41420-025-02470-4

The dual HDAC/PI3K inhibitor CUDC-907 inhibits ... - Nature CUDC-907, a novel dual inhibitor targeting the MYC upstream pathway (HDAC/PI3K), shows significant antitumor efficacy across multiple cancer types.

5. spandidos-publications.com spandidos-publications.com/10.3892/ijo.2023.5602

International Journal of Oncology - Spandidos Publications CUDC-907 is a dual-target inhibitor of PI3K and HDAC, which has significant potential to inhibit tumor growth and metastasis.

6. dana-farber.org dana-farber.org/clinical-trials/16-371

Phase 1 Trial of CUDC-907 in Children and Young Adults ... This research study is evaluating a novel drug called CUDC-907 as a possible treatment for resistant (refractory) pediatric solid tumors (including ...

7. thelancet.com thelancet.com/journals/lanonc/article/PIIS1470-2045%2815%2900584-7/abstract

Safety, tolerability, and preliminary activity of CUDC-907, a ... The safety and tolerability profile of CUDC-907 and the promising preliminary evidence of response support continued development of CUDC-907 ...